JP7256824B2 - 粒子状製剤用の凍結保護剤 - Google Patents

粒子状製剤用の凍結保護剤 Download PDF

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JP7256824B2
JP7256824B2 JP2020559465A JP2020559465A JP7256824B2 JP 7256824 B2 JP7256824 B2 JP 7256824B2 JP 2020559465 A JP2020559465 A JP 2020559465A JP 2020559465 A JP2020559465 A JP 2020559465A JP 7256824 B2 JP7256824 B2 JP 7256824B2
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composition
mrna
nano
cationic
therapeutically active
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JPWO2019207061A5 (pl
JP2021522248A (ja
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ドーメン，クリスティアン
ベック，フィリップ
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Ethris GmbH
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Ethris GmbH
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
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    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
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    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
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    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
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    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • A—HUMAN NECESSITIES
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    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—Organic compounds, e.g. phospholipids, fats
    • A—HUMAN NECESSITIES
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    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1682—Processes
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
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    • C12N2320/00—Applications; Uses
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  • Optics & Photonics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2020559465A 2018-04-25 2019-04-25 粒子状製剤用の凍結保護剤 Active JP7256824B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP18169325.0 2018-04-25
EP18169325 2018-04-25
EP18189010.4 2018-08-14
EP18189010 2018-08-14
PCT/EP2019/060646 WO2019207061A1 (en) 2018-04-25 2019-04-25 Cryoprotective agents for particulate formulations

Publications (3)

Publication Number Publication Date
JP2021522248A JP2021522248A (ja) 2021-08-30
JPWO2019207061A5 JPWO2019207061A5 (pl) 2022-04-28
JP7256824B2 true JP7256824B2 (ja) 2023-04-12

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JP2020558887A Pending JP2021522228A (ja) 2018-04-25 2019-04-25 Ｒｎａの送達用の脂質に基づく製剤
JP2020559465A Active JP7256824B2 (ja) 2018-04-25 2019-04-25 粒子状製剤用の凍結保護剤

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JP2020558887A Pending JP2021522228A (ja) 2018-04-25 2019-04-25 Ｒｎａの送達用の脂質に基づく製剤

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Country Link
US (2) US20210137840A1 (pl)
EP (3) EP4311541A3 (pl)
JP (2) JP2021522228A (pl)
KR (2) KR20210021943A (pl)
CN (2) CN112153985B (pl)
AU (2) AU2019258678B2 (pl)
BR (2) BR112020021054A2 (pl)
CA (2) CA3098262A1 (pl)
ES (2) ES2960936T3 (pl)
MX (2) MX2020011167A (pl)
PL (1) PL3784285T3 (pl)
WO (2) WO2019207060A1 (pl)
ZA (1) ZA202006026B (pl)

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TR201901396T4 (tr) 2013-03-14 2019-02-21 Translate Bio Inc Mesajcı rna'nın başlık verimliliğine ilişkin kantitatif değerlendirme.
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EA201690588A1 (ru) 2013-10-22 2016-09-30 Шир Хьюман Дженетик Терапис, Инк. Доставка мрнк в цнс и ее применение
CN105813656B (zh) 2013-10-22 2021-01-15 夏尔人类遗传性治疗公司 用于递送信使rna的脂质制剂
CA2949106C (en) 2014-05-30 2023-10-24 Shire Human Genetic Therapies, Inc. Biodegradable lipids for delivery of nucleic acids
JP6767976B2 (ja) 2014-12-05 2020-10-14 トランスレイト バイオ， インコーポレイテッド 関節疾患の治療のためのメッセンジャーｒｎａ治療法
EP3538068A1 (en) 2016-11-10 2019-09-18 Translate Bio, Inc. Improved ice-based lipid nanoparticle formulation for delivery of mrna
HUE059025T2 (hu) 2017-02-27 2022-10-28 Translate Bio Inc Módszerek a hírvívõ RNS tisztítására
MA49393A (fr) 2017-06-12 2020-04-22 Translate Bio Inc Poly(phosphoesters) destinés à l'administration d'acides nucléiques
US11167043B2 (en) 2017-12-20 2021-11-09 Translate Bio, Inc. Composition and methods for treatment of ornithine transcarbamylase deficiency
ES2960936T3 (es) * 2018-04-25 2024-03-07 Ethris Gmbh Formulaciones a base de lípidos para el suministro de ARN
JP7384832B2 (ja) 2018-05-16 2023-11-21 トランスレイト バイオ， インコーポレイテッド リボースカチオン性脂質
JP7488193B2 (ja) 2018-05-24 2024-05-21 トランスレイト バイオ， インコーポレイテッド チオエステルカチオン性脂質
CA3100214A1 (en) 2018-05-30 2019-12-05 Translate Bio, Inc. Cationic lipids comprising a steroidal moiety
WO2020035460A1 (en) * 2018-08-14 2020-02-20 Ethris Gmbh Lipid-based formulations containing salts for the delivery of rna
CN118421617A (zh) 2018-08-24 2024-08-02 川斯勒佰尔公司 用于纯化信使rna的方法
AU2020206109A1 (en) 2019-01-07 2021-07-29 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
SG11202112350XA (en) 2019-05-15 2021-12-30 Translate Bio Inc Methods for purification of messenger rna
WO2021072172A1 (en) 2019-10-09 2021-04-15 Translate Bio, Inc. Compositions, methods and uses of messenger rna
US20230399390A1 (en) 2019-10-24 2023-12-14 Novago Therapeutics Ag Novel anti-nogo-a antibodies
JP2023508881A (ja) 2019-12-20 2023-03-06 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａ担持脂質ナノ粒子を調製する改善されたプロセス
EP4110296A1 (en) 2020-02-25 2023-01-04 Translate Bio, Inc. Improved processes of preparing mrna-loaded lipid nanoparticles
WO2021245184A1 (en) 2020-06-02 2021-12-09 Neurimmune Ag HUMAN ANTIBODIES AGAINST SEVERE ACUTE RESPIRATORY SYNDROME CORONAVIRUS-2 (SARS-CoV-2)

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